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The antisense oligonucleotide rugonersen has acceptable safety and tolerability in children with Angelman syndrome and shows partial normalization of electroencephalogram abnormalities and clinical improvements relative to natural history.

A single-cell-resolution reference atlas of circulating hematopoietic stem and progenitor cells from healthy people reveals variations in cell-population frequencies and transcriptional profiles associated with age and sex. This atlas enables non-invasive diagnosis, risk stratification and follow-up of hematological conditions using peripheral blood, offering a potential alternative to bone marrow sampling.

In the dose-expansion cohort of this phase 1b trial, the EGFR×HER3 bispecific antibody–drug conjugate BL-B01D1 given to patients with metastatic esophageal squamous cell carcinoma led to encouraging clinical response rates, which need to be confirmed in larger efficacy trials.

In a phase 1 trial, treatment of individuals with mixed hyperlipidemia with a monoclonal antibody targeting the angiopoietin-like protein 3/8 complex resulted in dose-dependent reductions in the levels of triglycerides and low-density lipoprotein cholesterol, while increasing the levels of high-density lipoprotein.

In a silent trial involving 197 patients with lung cancer over a span of 4 months, a fine-tuned pathology foundation model was able to identify the presence of EGFR mutations with high accuracy, potentially preventing further genetic testing in 43% of cases.

In a large-scale proteomic study of biological aging of 11 organs from 44,498 individuals in the UK Biobank, the biological ages of the brain and immune system emerged as strong predictors of healthspan and longevity.

Siltartoxatug, a first-in-class recombinant monoclonal antibody for tetanus prophylaxis, was shown to be safe and superior to plasma-derived human tetanus immunoglobulin in achieving anti-tetanus neutralizing antibody titers 12 hours after administration.

Global estimates of expected and preventable gastric cancers in people born between 2008 and 2017 report that there will be 15.6 million lifetime gastric cancer cases, three-quarters of which are preventable through H. pylori eradication measures.

When tested in a multiethnic cohort of ~2.5 million US veterans, the PREVENT equations, recently developed by the American Heart Association for sex-specific and race-free prediction of cardiovascular disease risk, were shown to accurately estimate this risk with some variability across race and ethnicity groups and to outperform the previously used pooled cohort equation risk score.

Instead of making one-off treatments, industrialized gene therapy might be able to treat multiple diseases.

An explosion of digital tools and AI is catalyzing innovations in the mission to scale up mental health support worldwide. These paradigms currently rely on a ‘human in the loop’ — but who is the human in question, and does it matter?

Campaigners want ultra-processed foods to be better defined and regulated. But does the evidence support strong policy changes?

Preliminary results from an investigator-initiated clinical trial showed that an AAV-OTOF gene therapy was safe and led to hearing improvements in ten patients with congenital deafness with 6–12 months of follow-up, including in a teenager and a young adult, expanding the age range from previous trials.

A randomized phase 2a clinical trial of an AI-discovered drug and target combination for idiopathic pulmonary fibrosis shows safety and signs of efficacy, marking a concrete step forward in bringing AI-enabled drug discovery into the clinic.

A new analysis supports dietary guidelines to reduce the consumption of processed meat, sugar-sweetened beverages and trans-fatty acids — highlighting the need for a collaborative, meticulous health assessment framework for ultra-processed foods.